| Summary: | <p>Abstract</p> <p>Background</p> <p>The 6-minute walk test evaluates the effect of pharmacologic intervention in adults with pulmonary arterial hypertension (PAH) but, for reasons of compliance or reliability, may not be appropriate for children at all ages. Thus, peak oxygen consumption (VO<sub>2</sub>, maximal exercise test) was used instead in a pediatric PAH trial (STARTS-1) to evaluate pharmacologic intervention with sildenafil. This was the first large placebo-controlled trial to use the peak VO<sub>2</sub> endpoint in this population. Our working hypothesis was that, as with other populations, percentage changes in peak VO<sub>2</sub> in pediatric patients with PAH are reliable and are associated with changes in other clinical endpoints.</p> <p>Methods</p> <p>Using data from the subpopulation of 106 patients who were developmentally and physically able to perform exercise testing, all of whom were World Health Organization Functional Class (WHO FC) I, II, or III, reliability was assessed using the intraclass correlation coefficient and Bland-Altman plot on screening and baseline data. Relationships between percentage change in peak VO<sub>2</sub> from baseline to end of treatment and other endpoints were evaluated using correlation coefficients and regression analyses.</p> <p>Results</p> <p>The intraclass correlation was 0.79 between screening and baseline peak VO<sub>2</sub>, an agreement that was supported by the Bland-Altman plot. Percentage change in peak VO<sub>2</sub> correlated well (<it>r</it> ≥0.40) and showed responsiveness to a physician global assessment of change and with change in WHO FC (for baseline classes I and III). Percentage change in peak VO<sub>2</sub> did not correlate with change in the Family Cohesion of the Child Health Questionnaire (<it>r</it> = 0.04) or with a subject global assessment of change (<it>r</it> = 0.12). The latter may have been influenced by child and parental-proxy response and instrument administration.</p> <p>Conclusion</p> <p>In pediatric PAH patients who are developmentally and physically able to perform exercise testing, peak VO<sub>2</sub> measurements exhibited good reliability and improvements that were associated with improvements in certain other clinical endpoints, such as the WHO FC and a physician global assessment.</p> <p>Trial registration</p> <p>ClinicalTrials.gov identifier NCT00159913.</p>
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